NIH. US: Three-year
outcomes from an ongoing clinical trial suggest that high-dose
immunosuppressive therapy followed by transplantation of a person's own
blood-forming stem cells may induce sustained remission in some people
with relapsing-remitting multiple sclerosis (RRMS).
RRMS is the most
common form of MS, a progressive autoimmune disease in which the immune
system attacks the brain and spinal cord. The trial is funded by the
National Institute of Allergy and Infectious Diseases (NIAID), part of
the National Institutes of Health, and conducted by the NIAID-funded Immune Tolerance Network (ITN) .
Three years after the treatment, called high-dose
immunosuppressive therapy and autologous hematopoietic cell transplant
or HDIT/HCT, nearly 80 percent of trial participants had survived
without experiencing an increase in disability, a relapse of MS
symptoms or new brain lesions. Investigators observed few serious early
complications or unexpected side effects, although many participants
experienced expected side effects of high-dose immunosuppression,
including infections and gastrointestinal problems. The three-year
findings are published in the Dec. 29, 2014, online issue of JAMA
Neurology.
“These promising results support the need for future
studies to further evaluate the benefits and risks of HDIT/HCT and
directly compare this treatment strategy to current MS therapies,” said
NIAID Director Anthony S. Fauci, M.D. “If the findings from this study
are confirmed, HDIT/HCT may become a potential therapeutic option for
people with this often-debilitating disease, particularly those who
have not been helped by standard treatments.”
Scientists estimate that MS affects more than 2.3
million people worldwide. Symptoms can vary widely and may include
disturbances in speech, vision and movement. Most people with MS are
diagnosed with RRMS, which is characterized by periods of relapse or
flare up of symptoms followed by periods of recovery or remission. Over
years, the disease can worsen and shift to a more progressive form.
In the study, researchers tested the effectiveness of
HDIT/HCT in 25 volunteers with RRMS who had relapsed and experienced
worsened neurological disability while taking standard medications.
Doctors collected blood-forming stem cells from participants and then
gave them high-dose chemotherapy to destroy their immune systems. The
doctors returned the stem cells to the participants to rebuild and
reset their immune systems.
“Notably, participants did not receive any MS drugs
after transplant, yet most remained in remission after three years,”
said Daniel Rotrosen, M.D., director of NIAID’s Division of Allergy,
Immunology and Transplantation. “In contrast, other studies have shown
that the best alternative MS treatments induce much shorter remissions
and require long-term use of immunosuppressive drugs that can cause
serious side effects.”
The study researchers plan to follow participants for a
total of five years, recording all side effects associated with the
treatment. Final results from this and similar studies promise to help
inform the design of larger trials to further evaluate HDIT/HCT in
people with MS.
The work was sponsored by NIAID, NIH, and conducted by
the ITN (contract number N01 AI015416) and NIAID-funded statistical and
clinical coordinating centers (contract numbers HHSN272200800029C and
HHSN272200900057C). The ClinicalTrials.gov identifier for the study
High-Dose Immunosuppression and Autologous Transplantation for Multiple
Sclerosis (HALT-MS) is NCT00288626.
NIAID conducts and supports research — at NIH,
throughout the United States, and worldwide — to study the causes of
infectious and immune-mediated diseases, and to develop better means of
preventing, diagnosing and treating these illnesses. News releases,
fact sheets and other NIAID-related materials are available on the
NIAID Web site at http://www.niaid.nih.gov.
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Services. NIH is the primary federal agency conducting and supporting
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diseases. For more information about NIH and its programs, visit www.nih.gov.